The Food and Drug Administration (FDA) has expanded the indication for Kalydeco (ivacaftor; Vertex) to include treatment of cystic fibrosis in patients 6 months of age and older who have 1 mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. Previously, the treatment was not indicated for patients younger than 12 months old.

The approval was based on data from a phase 3 open-label safety study (ARRIVAL) which included a cohort of 11 children 6 months to <12 months of age. Findings from the study showed that the safety profile of Kaydeco was similar to that seen in older children and adults. The most common adverse reactions reported were cough, nasal congestion, and rhinorrhea. Following 24 weeks of treatment, the mean absolute change from baseline in sweat chloride (a biomarker) for patients aged 6 months to <12 months (N=6) was -58.6 mmol/L (95% CI; -75.9, -41.3).

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“Today’s approval for Kalydeco allows physicians to begin treating the underlying cause of CF in eligible infants as young as 6 months of age for the first time, with the potential to modify the course of the disease,” said Margaret Rosenfeld, MD, MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.

Kalydeco, a CFTR potentiator, is available as 150mg tablets for patients aged ≥6 years, and as 25mg, 50mg and 75mg oral granules for patients aged 6 months to <6 years

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